Health care resource utilization in the management of patients with Arginase 1 Deficiency in the US: a retrospective, observational, claims database study

Figures & data

Table 1. Baseline demographic characteristics.

	Pre-match			Post-match
	ARG1-D	Non-ARG1-D	p-Value	ARG1-D	Non-ARG1-D	p-Value
	(n = 77)	(n = 78,541)		(n = 77)	(n = 77)
Age
Mean	25.6	48.4	<.0001	25.6	26.3	.2407
SD	23.9	24.3		23.9	24.1
Median	15	53	<.0001	15	15	.2357
Age group (n, %)
0–1	6 (7.8)	1,233 (1.6)	<.0001	6 (7.8)	6 (7.8)	1.0
2–9	20 (26.0)	5,708 (7.3)		20 (26.0)	20 (26.0)
10–17	14 (18.2)	5,403 (6.9)		14 (18.2)	14 (18.2)
18–34	15 (19.5)	10,932 (13.9)		15 (19.5)	15 (19.5)
35–44	4 (5.2)	7,707 (9.8)		4 (5.2)	4 (5.2)
45–54	5 (6.5)	9,907 (12.6)		5 (6.5)	5 (6.5)
55–64	5 (6.5)	13,113 (16.7)		5 (6.5)	5 (6.5)
≥65	8 (10.4)	24,538 (31.2)		8 (10.4)	8 (10.4)
Sex (n, %)
Male	40 (51.9)	32,137 (40.9)	.0491	40 (51.9)	40 (51.9)	1.0
Female	37 (48.1)	46,404 (59.1)		37 (48.1)	37 (48.1)

Abbreviations. ARG1-D, Arginase 1 Deficiency; SD, standard deviation.

Figure 1. Prevalent manifestations. Abbreviation. ARG1-D, Arginase 1 Deficiency.

Table 2. Baseline clinical characteristics.

	ARG1-D	Non-ARG1-D	p-Value
	(n = 77)	(n = 77)
CCI (mean, SD, median)
Mean	1.1	0.3	.0015
SD	2.2	0.7
Median	0	0	.0008
CCI group (n, %)			.0698
0	54 (70.1)	62 (80.5)
1	8 (10.4)	11 (14.3)
2	3 (3.9)	3 (3.9)
3+	12 (15.6)	1 (1.3)
Manifestations of interest (n, %)
Spasticity	7 (9.1)	1 (1.3)	.0339
Developmental delay	7 (9.1)	4 (5.2)	.3657
Intellectual disability	5 (6.5)	1 (1.3)	.1025
Seizure	8 (10.4)	1 (1.3)	.0196
Other manifestations (n, %)
Abnormal gait	6 (7.8)	2 (2.6)	.1573
Failure to thrive/short stature	21 (27.3)	1 (1.3)	<.0001
Abnormal movement	3 (3.9)	0 (0.0)	.0833
Muscle stiffness	4 (5.2)	1 (1.3)	.1797
Hyperammonemia	18 (23.4)	0 (0.0)	<.0001
Comorbidities of interest (n, %)
Cerebral palsy	10 (13.0)	0 (0.0)	.0016
Liver disease	13 (16.9)	0 (0.0)	.0003
Cirrhosis/fibrosis	6 (7.8)	0 (0.0)	.0143
Epilepsy	15 (19.5)	2 (2.6)	.0003
GI conditions	19 (24.7)	10 (13.0)	.0606
Growth deficit	5 (6.5)	1 (1.3)	.1025
Depression	3 (3.9)	4 (5.2)	.6547
Anxiety	2 (2.6)	4 (5.2)	.4142
ADHD/behavioral disorders	3 (3.9)	6 (7.8)	.3173
Frequent comorbidities (n, %)
Disorder of urea cycle metabolism, unspecified	18 (23.4)	0 (0.0)	<.0001
Unspecified cirrhosis of liver	6 (7.8)	0 (0.0)	.0143
Hepatic failure, unspecified without coma	6 (7.8)	0 (0.0)	.0143
Medical procedures (n, %)
Physical/occupational therapy	12 (15.6)	7 (9.1)	.2513
CT	10 (13.0)	0 (0.0)	.0016
Complete blood counts with white blood cell differential	29 (37.7)	16 (20.8)	.0236
Routine venipuncture	23 (29.9)	17 (22.1)	.2888
Concomitant medications of interest (n, %)
Nitrogen scavenger
Sodium phenylbutyrate	4 (5.2)	0 (0.0)	.0455
Glycerol phenylbutyrate	2 (2.6)	0 (0.0)	.1573
Antispasmodic agent
Baclofen	10 (13.0)	1 (1.3)	.0067
Anti-epileptics	28 (36.4)	10 (13.0)	.0002
Medical foods	5 (6.5)	0 (0.0)	.0253
Psychiatric medications	21 (27.3)	14 (18.2)	.1779

Abbreviations. ARG1-D, Arginase 1 Deficiency; CCI, Charlson Comorbidity Index; SD, standard deviation.

Table 3. All-cause health care resource utilization.

	Pre-index			Post-index
	ARG1-D	Non-ARG1-D	p-Value	ARG1-D	Non-ARG1-D	p-Value
	(n = 77)	(n = 77)		(n = 77)	(n = 77)
Outpatient pharmacy
Patients with ≥1 prescription fill (n, %)	74 (96.1)	68 (88.3)	.0833	66 (85.7)	64 (83.1)	.6831
Patients with ≥1 prescription fill for glycerol phenylbutyrate, sodium phenylbutyrate, or sodium benzoate/sodium phenylacetate (n, %)	6 (7.8)	0 (0.0)	.0143	4 (5.2)	0 (0.0)	.0455
Patients with ≥1 prescription fill for other ARG1-D-related medications (n, %)	4 (5.2)	0 (0.0)	.0455	2 (2.6)	0 (0.0)	.1573
Outpatient medical
ER visits
Patients with ≥1 ER visit (n, %)	27 (35.1)	15 (19.5)	.0285	27 (35.1)	14 (18.2)	.0326
Number of ER visits per patient (mean, SD, median)
Mean	0.8	0.3	.0034	0.6	0.4	.1819
SD	1.6	0.7		1.1	1.0
Median	0.0	0.0	.0024	0.0	0.0	.1048
Physician office visits
Patients with ≥1 physician office visit (n, %)	44 (57.1)	56 (72.7)	.0339	44 (57.1)	58 (75.3)	.0164
Number of physician office visits per patient (mean, SD, median)
Mean	2.8	2.6	.8031	3.4	2.8	.4842
SD	5.0	3.7		6.0	3.5
Median	1.0	2.0	.6780	1.0	1.0	1.0000
Laboratory and pathology tests
Patients with ≥1 laboratory and/or pathology test (n, %)	46 (59.7)	37 (48.1)	.1495	50 (64.9)	33 (42.9)	.0065
Number of laboratory and/or pathology tests (mean, SD, median)
Mean	8.7	4.4	.1042	6.8	2.9	.0026
SD	19.7	11.4		8.2	6.8
Median	1.0	0.0	.0075	3.0	0.0	<.0001
Inpatient stay
Valid n (%)	17 (22.1)	16 (20.8)	.8443	17 (22.1)	16 (20.8)	.8443
Patients with ≥1 inpatient stay (n, %)	5 (29.4)	2 (12.5)		3 (17.6)	1 (6.3)
Number of inpatient stays per patient (mean, SD, median)
Mean	0.4	0.1	.1546	0.4	0.1	.2663
SD	0.7	0.3		1.0	0.3
Median	0.0	0.0		0.0	0.0
Length of stay per patient for all inpatient stays (mean, SD, median)
Mean	5.2	0.3	.0882	2.4	0.3	.1887
SD	11.1	0.9		6.4	1.0
Median	0.0	0.0		0.0	0.0

Abbreviations. ARG1-D, Arginase 1 Deficiency; ER, emergency room; SD, standard deviation.